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PTC Therapeutics Announces Results of Phase 3 Study of Potential CF Drug Ataluren

June 8, 2012

PTC Therapeutics Inc. today announced results from a Phase 3 clinical trial of ataluren, a potential oral drug aimed at treating the underlying cause of cystic fibrosis.

The 48-week study tested ataluren in people with CF ages 6 and older who have what is known as a “nonsense mutation.”

The results showed that study participants who received ataluren had a lower decline in lung function and a lower rate of pulmonary exacerbations, compared with those who took a placebo.

PTC Therapeutics said it will be discussing the data with its clinical researchers and with advocacy groups to determine the next steps.

About 10 percent of people with CF have nonsense mutations, which interrupt the production of the CFTR protein — the key protein associated with CF — causing it to be too short and not function normally. Ataluren is designed to enable the production of a full-length and functional CFTR protein.

The Phase 3 study enrolled more than 230 volunteers at multiple sites in North America, Europe and Israel.

The Cystic Fibrosis Foundation provided support for the development of ataluren.

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